US approves trials with gene therapy

HUMAN gene therapy begins this autumn in the US after years of research to prove that it is safe and potentially effective. But new strategies for using genes against disease could soon make these first attempts obsolete.

Last week, a panel of experts at the National Institutes of Health ushered in the age of gene therapy by approving two experiments conceived more than three years ago. In one, children with a defective gene that cripples their immune systems will receive new working versions of the gene incorporated into their white blood cells. The new genes should create a missing enzyme, adenosine deaminase (ADA). Without the enzyme, the children eventually die of infections.

In the second experiment, Steven Rosenberg of the National Cancer Institute will draw white blood cells called tumour infiltrating lymphocytes (TIL) from patients with terminal skin cancer. The gene added to these cells should in essence supercharge them, ...

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